low-dose baricitinib plus high-dose dexamethasone for patients with newly diagnosed immune thrombocytopenia

Conditions: Immune Thrombocytopenia

Summary: a randomized, open-label, multicenter, phase 2 trial to compare the efficacy and safety of baricitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (itp).

this is a parallel group, multicenter, randomized, controlled trial of patients with itp in china. patients were randomly assigned to receive baricitinib plus high-dose dexamethasone or high-dose dexamethasone monotherapy. treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request. the primary endpoint is durable response, defined as the maintenance of platelet count ≥30,000/μl and at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

Eligibility criteria:
inclusion criteria: 1. confirmed newly-diagnosed, treatment-naive itp; 2. a platelet count \<30,000/μl, or a platelet count \<50,000/μl with clinically significant bleeding symptoms (who bleeding scale 2 or above) at the enrollment; 3. willing and able to sign written informed consent. exclusion criteria: 1. received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit; 2. have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct coombs test; 3. active or a history of malignancy; 4. pregnancy or lactation; 5. received first-line and second-line itp-modifying therapy; 6. previously received corticosteroids or immunosuppressive agents for non-itp diseases within 6 months before enrollment; 7. a history of clinically significant adverse reactions to previous corticosteroid therapy; 8. unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia); 9. current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection; 10. a history of symptomatic herpes zoster infection within 12 weeks prior to screening; 11. active or chronic viral infection from hepatitis b virus (hbv), hepatitis c virus (hcv), or human immunodeficiency virus (hiv); 12. have evidence of active tuberculosis (tb), or have previously had evidence of active tb and did not receive appropriate and documented treatment, or have had household contact with a person with active tb and did not receive appropriate and documented prophylaxis for tb; 13. have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled; 14. myocardial infarction (mi), unstable ischemic heart disease, stroke, or new york heart association stage iv heart failure; 15. a history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data; 16. any of the following specific abnormalities on screening laboratory tests: 1) alt or ast \>2 x uln, or total bilirubin ≥1.5 x uln 2) hemoglobin \<9 g/dl, or total white blood cell (wbc) count \<2,500/µl, or neutropenia (absolute neutrophil count \<1,200/µl), or lymphopenia (lymphocyte count \<750/µl) 3) egfr \<50 ml/min/1.73 m\^2.

Keywords: baricitinib high-dose dexamethasone